Editas Medicine, Inc. (EDIT) Presents at 41st Annual J.P. Morgan Healthcare Conference (Transcript)

Editas Medicine, Inc. (NASDAQ:EDIT) 41st Annual J.P. Morgan Healthcare Conference January 10, 2023 7:30 PM ET

Company Participants

Gilmore O’Neill – Chief Executive Officer

Baisong Mei – Chief Medical Officer

Michelle Robertson – Chief Financial Officer

Conference Call Participants

Brian Cheng – JPMorgan

Gilmore O’Neill

Okay. Good afternoon, everyone. I am being told to progress, to maintain the clock. And I also understand that I am the last speaker between you and the bar. So even more important that I get this right and follow instructions. Brian, forgive me. I started without you. Sorry.

Brian Cheng

Not a problem.

Gilmore O’Neill

I was under instructions, Brian. Okay. So today, Editas is changing. Editas is changing so that we can be the leader in in vivo programmable gene editing leveraging cutting-edge technology. We aim to deliver therapies that revolutionize the treatment of previously untreatable diseases, improve patients’ treatment journeys by simplifying the usability of gene editing and minimize the burden on health care systems, all while developing novel, differentiated medicines.

Today, I am pleased to share our strategy to achieve this vision. The underlying pillars of the strategy are: an expanded focus on hemoglobinopathies and in vivo editing, a strengthened discovery engine focused on in vivo gene editing in multiple cell types and tissues and an increase in business development efforts. And we are well on our way towards this vision as we’ve begun to execute these changes. In the near term, Editas would expand its focus on hemoglobinopathies in 3 ways, building on our recent clinical success with our EDIT-301 program. We will continue to advance our ex vivo 301 program in sickle cell anemia and beta thalassemia. We will pursue milder conditioning regimes to lessen the burden and risks suffered by patients on their clinical journey, and we will pursue in vivo editing of hematopoietic stem cells.

Now during today’s presentation, I will make forward-looking statements. Actual results might differ materially from those projected in such statements. So please refer to our federal securities filings for additional information.

But let me pause for a second and introduce Editas to those who are less familiar with us. Editas is a clinical-stage gene editing pioneer that has built a cutting-edge biotech platform in order to develop differentiated medicines for patients with previously untreatable or inadequately treated diseases. The company was formed nearly 10 years ago and since then has driven our technology forward across a range of in vivo and ex vivo programs, and this is where this pioneering work has brought us to today.

Editas has important strengths that provide building blocks for the transformation that we are undertaking. And these strengths include Editas’ core technology that includes a differentiated high-fidelity AsCas12a CRISPR nuclease with the ability to delete and/or correct disease-causing mutations in clinical application, core expertise in guide RNA design and chemistry, advanced bioinformatics and well-developed CMC quality and analytics. It also includes a strong IP portfolio covering Cas9 and AsCas12a and a set of human proof of concept readouts in the past 3 months. AsCas12a is our proprietary high-fidelity, high-specificity nuclease and has demonstrated superior characteristics that increase the efficiency of editing and significantly reduce off-target editing seen with other nucleases. Moreover, preliminary data from our RUBY study suggests we have achieved human proof of concept utilizing AsCas12a in our EDIT-301 program.

I joined Editas because I saw a huge opportunity to help transform Editas from a technology platform company into a commercial therapeutics company. I was appointed as CEO 6 months ago with a few near-term objectives: to hire a world-class Chief Medical Officer, to sharpen our focus on clinical execution and to refine Editas’ strategy. I am pleased with our progress on these 3 objectives to date.

First, in July, we hired Baisong Mei, our Chief Medical Officer. Baisong brings over 20 years of experience in the biopharma industry and most recently served as Senior Global Project Head for Rare Disease and Rare Blood Disorders at Sanofi. Baisong and I are delighted at the opportunity to work together again as we had worked in the past at Biogen together. Second, we have driven clinical execution to 2 clinical readouts in the past 3 months. And third, we are ready to share Editas’ vision, strategy and structure for the future and the objectives that we have set for 2023 on our path to realizing our new vision, a vision where Editas would be the leader in in vivo gene editing.

As I stated a few minutes ago, we have recently generated human proof of concept data in 2 clinical programs and made milestone decisions on both. First, less than 2 months ago, we announced human proof of concept for our in vivo EDIT-101 AAV-delivered Cas9 therapy for LCA10, or Leber congenital amaurosis 10, an inherited retinal disease. However, due to the limited addressable population size, we made the tough decision to pause enrollment in our BRILLIANCE trial until we found a partner.

Second, one month ago, we shared very encouraging initial data from the RUBY Phase I/II clinical study of our ex vivo autologous EDIT-301 therapy in severe sickle cell disease. The readout suggested human proof of concept that EDIT-301 could safely drive expression of fetal hemoglobin to clinically meaningful levels and correct anemia in sickle cell disease patients. It also demonstrated that Editas has a potential product that can give robust clinical benefit to patients and has the potential for clinical differentiation in the long term. With our current technological strength and these clinical data, Editas is poised to redefine its vision and strategic priorities.

Now where is Editas going as a gene editing clinical-stage therapeutics company with a clinically validated technology? We are changing. And our long-term vision, to be a leader in in vivo programmable gene editing that leverages cutting-edge technology to deliver therapeutics that includes the simplifying the usability of impact — and impact, excuse me, of its therapies by minimizing the burden to patients and health care systems and to avoid a me-too mindset by selecting therapeutic targets where Editas can meaningfully differentiate from the current standard of care.

So how will we do this? First, we are narrowing the focus of our discovery to in vivo-administered genome editing medicines while continuing to develop our existing ex vivo drug candidate for sickle cell disease and transfusion-dependent thalassemia. We will no longer discover or develop stand-alone cell therapies, and we’ll seek to divest stand-alone allogeneic NK oncology assets. We will continue supporting our partnered cell therapy programs. This includes our T cell programs with both BMS and Immatics. And additionally, we have terminated our AAV IRD platform and seek to divest those assets.

Second, we are strengthening our discovery engine and capabilities. We have split our research division into separate technology and drug discovery groups, strengthening the capabilities of each. Our technology group will have a focus on targeted delivery starting with HSCs, or hematopoietic stem cells, and enhancing our gene editing toolbox to enable targeted gene repair. This technology group will be headed up by Bruce Eaton, a veteran technology entrepreneur, who will assume the role of Chief Technology Officer.

The drug discovery group will initially focus on in vivo therapeutic target selection in hematopoietic stem cells. We are also looking beyond HSCs, or hematopoietic stem cells, at other cell types and tissues and look forward to sharing updates in the future. We will select therapeutic targets we believe have a significant probability of technical, regulatory and commercial success. And with the change in the structure and our move away from AAV IRD, we have initiated a search for a new CSO. Mark Shearman, our current CSO and an AAV IRD expert, will support a smooth transition through the end of the first quarter of this year.

And third and finally, we are increasing our business development activities to complement our internal technological capabilities and to leverage our IP portfolio. This strategic focus — or refocusing allows us to concentrate on our talent. It has also reduced our headcount by 21% and increases our expected cash runway into 2025.

Moving to our development plans, the EDIT-301 data in sickle cell disease have shown us where we can start to be most transformative and effective. We will pursue a leadership position in HSC therapeutics for hemoglobinopathies, both ex vivo and in vivo delivery settings, by building off our foundational knowledge from EDIT-301. One of our key objectives will be to accelerate clinical development of this lead program, and specifically, we will add to our program by taking 3 actions.

First, we will increase our investment in editing capacity and enrollment of patients in our RUBY and EDITHAL trials as we continue to advance our ex vivo 301 program for sickle cell and beta thalassemia. Second, we will pursue milder conditioning regimes, improving the therapeutic proposition for our sickle cell and TDT patients. This should increase adoption of an EDIT-301 therapy by reducing or eliminating the acute burdens of immunosuppression at the time of transplant and reducing the long-term burdens of infertility and oncogenesis associated with current toxic conditioning regimes. And third, we will pursue in vivo editing of hematopoietic stem cells through enhanced targeted delivery of our AsCas12a nuclease through our clinically validated hemoglobin gamma 1/2 promoter site.

Before talking about the specifics of our EDIT-301 clinical program, I would like to provide a bit of background on sickle cell disease and the patient experience. Sickle cell disease affects millions of people worldwide, including approximately 100,000 people in the United States. Severe sickle cell disease is an inherited, life-threatening and disabling hematologic disorder manifesting shortly after birth and characterized by unpredictable and severe attacks of acute pain or vascular occlusive crisis, progressive organ damage, chronic anemia and shortened lifespan.

Patients with severe sickle cell disease experience daily challenges in addition to those painful vascular occlusive crises. Their anemia causes fatigue, low exercise tolerance and cardiopulmonary strain. They may also suffer strokes, MIs, renal damage, resulting in ultimate organ failure. Current treatment options for sickle cell patients are limited. Allogeneic bone marrow transplantation can cure sickle cell disease, but less than 20% of patients can find match donors and there’s a risk of serious complications beyond simply those of conditioning. Other approved treatments show limited effectiveness and do not address the underlying cause of the disease.

EDIT-301 seeks to increase the expression of fetal hemoglobin using our novel AsCas12a nuclease to edit the promoter regions of gamma globin genes 1 and 2 in patients’ HSCs. This unique approach mimics the natural mechanism of hereditary persistence of fetal hemoglobin, thereby reducing the probability of sickling of red blood cells in sickle cell patients.

We announced initial data for EDIT-301 last month with the preliminary data suggesting that Editas Medicine has a product that can give robust clinical benefit to patients with severe sickle cell disease and has the potential for clinical differentiation in the long term. In the first patient treated with EDIT-301, fetal hemoglobin fraction increased from 5% to 45.4% and total hemoglobin increased from 11.9 grams per deciliter to normal levels at 16 grams per deciliter 5 months after transplant. 96% of patient 1’s red blood cells express fetal hemoglobin, that is a ubiquitous expression, and mean corpuscular fetal hemoglobin levels reached 13.8 picograms per red cell after 5 months, thus reaching and exceeding a threshold that will prevent sickling of individual red cells.

We have confidence that we will continue to see these effects in our growing clinical patient series in RUBY. The clinical data from RUBY recapitulate our preclinical experience in both the magnitude of the fetal hemoglobin response and the distribution of fetal hemoglobin across red blood cells or F-cells. We expect EDIT-301 to be well positioned to capture a meaningful segment of sickle cell and thalassemia patients. We believe that EDIT-301 will be part of the evolving market for next-generation hemoglobinopathy medicines and that the vast majority of eligible patients will still be untreated, owing to slow uptake and reimbursement ramp at the time of our launch. We have seen this experience across multiple specialty and rare disease markets in the recent past.

We will increase our investment to grow our EDIT-301 editing capacity to support increased clinical trial enrollment and dosing now that all sentinel patients have been dosed in a graft. We already enrolled additional patients for the RUBY and EDITHAL trials and collected and edited their hematopoietic stem cells and are scheduling their transplants as we continue to screen new patients at current sites and activate new clinical trial sites.

Turning to in vivo. We will focus our technology on targeted in vivo editing. Our initial focus is on in vivo editing of HSCs, or hematopoietic stem cells. EDIT-301 ex vivo demonstrates successful hemoglobin induction and creates a strong foundation for that initial in vivo project. The ability to edit hematopoietic stem cells directly in patients without transplantation will allow the treatment of much larger patient populations. In fact, the majority of sickle cell and thalassemia patients has no myeloablative conditioning is required. Indeed, pheresis would actually also not be required. Such a product could be used across multiple health systems with a much lower burden on patients and treatment centers. Beyond HSC, we will also focus our discovery efforts on other tissues, and we look forward to updating you on additional areas of focus as our discovery efforts progress.

Now on the business development front, we will partner and collaborate to extend our reach, access, capabilities that are complementary to our current technology and accelerate programs as we drive our programs towards commercialization. We will continue to out-license EDIT’s — or Editas’ technology for promising areas. And we will also leverage our IP around Cas9 and AsCas12a to increase our BD activities where our strong IP position creates value directly and through additional BD opportunities.

As things stand today, Editas is the exclusive licensee of the Cas9 IP portfolio owned by the Broad Institute for human medicines. That is, any company that wants to commercialize a gene editing product in the U.S. as well as in other markets that uses the Cas9 enzyme for human therapeutics would need to obtain a license from Editas Medicine. Now with our narrowed focus, we plan on progressing EDIT-301 through clinical trials while currently advancing our earlier-stage hematology initiatives and other initiatives, as outlined earlier.

We look forward to executing on our strategy, continuing our transformation and sharing our progress with you. As we look more specifically at 2023, our strategic objectives for the year include: hiring a new CSO with specific expertise aligned to our vision, resetting our discovery group, initiating discovery of in vivo editing of HSCs and in other tissues. And on the development side, we plan to execute on the following: providing a clinical update from the EDIT-301 RUBY study in mid-2023, which will include the initial 2 patients that were dosed last year as well as additional patients from the ongoing RUBY trial. In addition, we will have dosed 20 patients in our EDIT-301 RUBY program by year-end. We will dose the first patient in EDIT-301 EDITHAL trial for thalassemia in the first quarter of this year, and we look forward to providing early data from our sentinel patients in the EDITHAL trial for TDT later this year.

Last year was a pivotal year in Editas Medicine’s history as we reset our vision, began execution on this vision and hit multiple milestones of progress for this changed Editas. And we expect this year to be even more eventful. Looking forward, we will deliver on Editas’ potential by resetting and strengthening our discovery platform and efforts to focus on in vivo editing using criteria that maximize the probability of technical, regulatory and commercial success; by increasing our investment in EDIT-301, enhancing the patient experience and looking beyond ex vivo therapy to in vivo editing of HSCs; and increasing and enhancing our business development activities to create value. It is really exciting to be part of this next chapter in Editas’ journey as we transform to deliver curative medicines to people living around the world with serious diseases. Thank you very much.

Brian Cheng

Okay. So we’ll switch over to Q&A session. I welcome Baisong, Michelle as well on the stage.

Question-and-Answer Session

Q – Brian Cheng

Welcome to the Q&A session. It turns out to be extremely difficult to go from session to session in the Westin. So maybe just to kick start the conversation. I think when we look at the gene editing space, in the last couple of months, we’ve seen a lot of debates on what the regulators think about gene editing in general, right? You see other gene editing companies having a little bit of a hurdle to get things done in terms of getting the first clinical studies in the U.S. cross-checked and started in the U.S. Maybe just one broad question to kick-start is to, maybe Gilmore can answer this, is how do you think about just the current receptivity from the regulatory agency on gene editing? And what is your strategies to — as you turn a new page on your portfolio, what’s your strategy that you think would be the best to get — advance your portfolio the fastest?

Gilmore O’Neill

Yes. Thanks, Brian. So obviously, I’m going to articulate some opinions about regulatory agencies, which should be seen as just that. The good news is that I have substantial experience in working with regulators around the world and with the FDA, and I’m actually joined by Baisong here, who also has substantial experience. And I think between the 2 of us, we have had 10 drugs approved and have seen all the vagaries of development challenges, discovery challenges and regulatory challenges.

So how are the regulators looking at gene editing if I think about it? Well, I think in many ways, we’ve actually also had a very good experience just in the last year if we look more comprehensively than through targeted CRISPR-based gene editing in that we have seen an approval for a lentiviral-edited therapeutic. And I think what that demonstrated is a regulatory agency that actually weighs very carefully, and obviously listens to important advice for the AdCom, but weighs very carefully benefit risk. And obviously, looking at or targeting diseases that are very serious, have significant morbidity, have an inadequate established standard of care for patient needs in the context of therapy with either yet to be defined or comprehensively defined risk or a well-defined risk, as we’ve already seen with lentiviral, the agencies and the FDA specifically are well able to actually approve those medicines. So I think that’s a very important point.

I think you touched on something else around some of the issues in recent months with other interactions with regulators. And I think the important piece there is that the agency quite rightly focuses very much on the creation and development and manufacturing of your clinical supply as well as, ultimately, your commercial supply. And that’s actually a reason why Editas upfront has invested significantly on both its research analytics for risk management as well as on its CMC analytics and quality. Those are — what I would say, they’re not as sexy as drug discovery, but they’re absolutely vital. They’re kind of the black box that no one wants to look at. Or as I said recently to somebody, when you’re flying an airplane, you’re looking at the air quality, how much seat room you have. But ultimately, the things that are actually keeping the thing in the air are the engines and it’s — they’re not visible to you, but they’re the things that keep the engines — or the plane in the air.

So I think the key point is to really, our priority, look forward to managing risk, and that includes both from a discovery point of view as well as really thinking about your manufacturing. Then from our point of view, our strategy, how we think about that, I’ve kind of actually hinted at that, which is select diseases that are very serious certainly at initial or certainly early on with this technology because then you can get your benefit-risk right.

I also think, frankly, that actually, while ultimately we want to expand the use of gene editing to very large patient populations, I think in the beginning, you have to think about managing risk in two ways: balancing the risk and benefit in individual patients; and then for regulators, recognizing they also have to consider the risk of a public health risk, which is releasing a new therapeutic where the safety profile is being — or is evolving into large populations. That is — those are two ways to actually manage that. I think Baisong and myself had significant experience in bringing newer technologies to market. I don’t know, Baisong, if you want to add to that.

Baisong Mei

No, I agree with you, Gilmore. I think as we are developing novel therapies, right, so I think the most important thing we are interested as well as regulatory agency interested is benefit-risk balance of that.

Brian Cheng

Okay.

Gilmore O’Neill

Can I just say one other thing? Because we’ve talked about risk a lot. I think this has actually been a really — last year was really a very interesting and exciting year for, I’d say, programmable or CRISPR-based gene editing because in that year and even in the year before, we have actually seen — in 10 years after the original publication of the use of CRISPR for eukaryotic cells, we’ve actually seen multiple proofs of concept with in vivo and ex vivo therapeutics from multiple sponsors. This is a phenomenal achievement, and I think it actually is the prelude to really exciting developments. Managing risk is very important, but managing risk when you have robust efficacy with the technology is much easier.

Brian Cheng

So just to follow up on that is when you think about your next step — you previously guided that you would disclose the strategic priorities and — which you did very recently, and thanks for giving us a recap on the priorities and how you envision the next year and beyond look like for an Editas. Maybe just to backtrack a little bit as to you have proof of concept in a few inherited retinal diseases. You also have proof of concept of 301. What gave you the confidence to push for 301? And you also have also divided your R&D into two divisions as well. Maybe just some thoughts on what is giving you the confidence that this will be the right strategy to move forward.

Gilmore O’Neill

I think there are a number of factors. I did outline, maybe not in sufficient detail, our approach to target selection and therapeutic selection. One, we just discussed a little bit about, was selecting very serious diseases with significant morbidity with inadequate standard of care. The other was ones which are clearly monogenic and where the genetics and the biology are really well understood. Indeed, if there’s a natural experiment that actually helps you define a likely dose response through allelic interrogation — allelic series interrogation, et cetera, even better. Wonderful examples of that are hereditary persistence of fetal hemoglobin and the SMA experience where you can actually even predefine therapeutic or biological therapeutic thresholds based on a natural experiment in humans.

The other is to look at translatability. And I think this actually feeds into one of the decisions where we’ve got IRDs or retinal dystrophies, which is the ability to test a biological parameter easily, repeatedly if necessary, and that biological parameter is very close to or proximal to the editing target. A very good example of that, for example, is fetal hemoglobin, which is easily sampled by venipuncture. Unfortunately, in contrast, no such assay — or robust assay exists for retinas today.

And then finally, obviously, looking at the regulatory path. And here, Baisong and myself, with the experience of bringing 10 drugs to approval, have a very good sense of what that looks like. You don’t have to have precedent, but you need to actually know what it looks like and actually have passed the reg phase test. And Baisong and I, I think, collectively have certainly experienced enough reg phase tests over the years to really know what that looks like. And then finally, make sure that this is commercially viable both from a competitive landscape and again, more importantly, around the established standard of care.

Brian Cheng

So just to hone in on this point, you laid out a few pillars as to how you think about the next step, right? One of them is commercial viability, translatability. And so we know that EDIT-301 is your key focus, but you also laid out the fact that you’re also looking into next-generation in vivo assets. Any color on how we should think about what would be, just the broad area that you’ll be focusing on, what would be an interesting disease that could be — could go well with what you have today?

Gilmore O’Neill

Well, I think the first in vivo therapeutic we’re going to actually zero in on is the editing and targeting of hematopoietic stem cells. Why is that actually a good idea? Well, we have actually validated by in-human proof of concept the target, which is the gamma globin promoter, and the mechanism for editing AsCas21a. What we now — it actually enables us to kind of reduce the magnitude of the problem to one of targeted delivery at — to hematopoietic stem cells in vivo. And we’ve actually started some work on that and obviously, we’ll need to continue.

One of the things that gives me confidence is other elements of our strategy. Unfortunately, I’m stealing, Michelle’s thunder by saying that as part of this effort, we actually have freed up cash, extended our cash runway and been able to redeploy our capital in ways that can actually critically help us advance beyond just 301, but on the in vivo side in growing and strengthening our internal efforts. But actually also philosophically, and this is another part of our strategic change, a philosophical change to actually embrace the idea of bringing in or partnering other technologies that will complement ours in actually delivering to the targeted tissue here, HSCs, with a view to developing one of our in vivo targets.

So that’s an example of a disease and the reasons why we have some confidence — or a lot of confidence there. Beyond that, I really want to bring in our CSO who will be partnering with Bruce Eaton, our CTO; and Baisong, our CMO, to really think very robustly about the targets that we will bring forward using the criteria that I’ve just outlined for selection, with a real focus on not being a me-too. And then we’ll be looking forward to sharing that with you in the future.

Brian Cheng

Okay. Maybe just turning over to EDIT-301. What are the next steps for the program? And maybe remind us — remind the audience, what is the bar for fetal hemoglobin that you’re shooting for and just the next steps moving forward for the program?

Gilmore O’Neill

Bar for — sorry, I misheard you.

Brian Cheng

The biomarker that you’re looking for.

Gilmore O’Neill

Yes. Forgive me. Yes. So our next steps, I’m going to sort of divide it into 2 pieces. I’ll talk a little bit about what we’re doing with regard to investment in editing capacity. So one of the things we’ve actually restarted with the redeployment of capital is actually grow our editing — our internal editing capacity, which includes both manufacturing and quality analytics, so that we can actually guarantee supply — clinical supply to the clinical program as that advances.

Quickly, I’ll say that the — one of the key biomarkers is fetal hemoglobin. Fetal hemoglobin actually speaks for itself. It’s the hemoglobin that circulates in our veins until we’re born, and then we switch it off and switch on adult hemoglobin. We do that by switching off gamma globin, switching on beta globin. Unfortunately, patients with sickle cells or thalassemia have mutations in the beta globin gene, and the natural experience of hereditary persistence of fetal hemoglobin has shown that switching back on fetal hemoglobin substantially mitigates the complications of disease. Indeed, in patients who are at risk of sickle cell disease, there seems to be a dose effect — or a dose-dependent effect on multiple complications well beyond VOCs. But with regard to the next steps with the clinical trial, I’d like to pass it to Baisong.

Brian Cheng

And what is the bar that you want to hit? Because it seems…

Gilmore O’Neill

The bar, okay, the level or the threshold, yes.

Brian Cheng

Yes, because I think we saw a couple of updates for fetal — in the same disease. I wonder if you have any updates on how you think about just what you want to show?

Gilmore O’Neill

Yes. Well, I think there are two things. One is that I talked about the hereditary persistence of fetal hemoglobin and sort of a number of academic analyses of the data suggest that you get a dose-dependent effect: higher fetal hemoglobin, better control of multiple complications of sickle cell disease. From a bar point of view, from the elimination of VOCs, it certainly looks in that experience that VOCs can be largely controlled or eliminated by hitting a bar of about 30% fetal hemoglobin. However, it is not unreasonable to expect that if you go higher, and certainly with our first patient, we’ve achieved levels of 45% fetal hemoglobin, you would anticipate more control and better outcomes, including for the control of other complications because as I said in the presentation, sickle cell disease is about much more than VOCs.

For those of you who are used to multiple sclerosis or have any experience there, multiple sclerosis has acute attacks, but an underlying chronic degenerative disease that ultimately causes incapacity. You can think rather loosely in similar terms that VOCs are acute attacks, extraordinary unpleasant and unpredictable attacks, but underlying that is a progressive vascular and vasculopathy and damage to multiple end organs: CNS, heart, kidney, et cetera. So actually, going higher, we believe, may actually have a real benefit from a bar point of view. And I think that’s important, I think, as you talk about other recent disclosures.

Brian Cheng

Right. Maybe, Baisong, you can shed light on what the next step is for the program?

Baisong Mei

Sure. Yes. As Gilmore mentioned earlier, one of the priority that Gilmore and I joined the company is to focus on clinical execution, right? So we’ve been working on rare disease for many years, and you have to reach out to patients. You have to reach out to community. You have to reach out to investigators. So with this effort by the team, really, we see big momentum in terms of patient enrollment. So we now have two patients dosed and have multiple patients enrolled but also have their cells collected and edited and ready for infusion for the transplantation of that.

So really, what we’re looking into that is we will have data released in middle of this year to have the data for these two patients — longer-term data for these two patients dosed as well as the data from multiple patients to be dosed in the next few months. So this is on the sickle cell side. And we also have huge momentum from the EDITHAL study, which is for beta thalassemia patients. And we also have quite a few patients enrolled already, actually have been processed, been — either already completed pheresis editing or in the process of doing that, and we plan to dose those patients very soon. And we also plan to release the data for EDITHAL patients end of this year. So we have huge momentum for the EDIT-301 study.

Gilmore O’Neill

And then to reiterate our specific target or objective for this year, we will have dosed 20 patients in the RUBY study by the end of the year. Just want to be sure that…

Brian Cheng

Yes. Maybe just on the preconditioning regimen that you’re working on. How does that fit into — does that have any implication in the RUBY trial? Or are you thinking about working on it in a separate study?

Gilmore O’Neill

Well, let me just say, and then Baisong can talk more, what I want to say is just from a strategic point of view, the intent is to drive 301 to approval as rapidly as possible. The milder conditioning regime enables us to expand the availability of the medicine upon its approval. So it doesn’t necessarily have to be developed at the same time. Essentially, it’s going to be, we believe, an important evolution, as do others, in stem cell transplantation in general and specifically for sickle cell and thalassemia because patients who would not otherwise be eligible, for example, for busulfan conditioning would be eligible and be able to tolerate a milder conditioning just because of general bone marrow health or other health issues.

So you should look at it more as a step. Whereas we go for 301 approval, the next step is to expand use and availability by milder conditioning. But the ultimate or, I’d say, the omega point, the real target or north star is to actually get to in vivo editing where you can essentially eliminate a substantial amount of risk that has nothing to do with the therapeutic. It’s more tied to the conditioning. You can reduce the consumption of health care resources by eliminating the need for pheresis, for isolation units, et cetera. And obviously, you make the risks and the tolerability of the therapy much greater for patients. Baisong, I think with regard to your thoughts about developing…

Baisong Mei

No. I think well said, Gilmore. I think from clinical development strategy perspective, we really wanted to push this EDIT-301 to the finish line, right? But we are taking a parallel approach to really also look into the milder conditioning, and this milder conditioning, in terms of channel strategy wise, is not going to be just for sickle cell alone, right? It’s going to be beneficial for many different things in there. So there will be really a development for its own and will benefit for EDIT-301 as well as other potentially, transplant wise.

Brian Cheng

Maybe one question for Michelle. How does — the strategic reprioritization that you announced earlier, how does that impact your expenses moving forward? And as you think about new programs rolling into your portfolio, how does that change your cash runway?

Michelle Robertson

Sure. So as Gilmore mentioned, the narrower focus in the strategy has resulted in our cash runway extension into 2025. This is important for a couple of reasons. The first one is that it’s going to allow us to fully invest in 301 as we move it through to the value inflection points: the accelerated enrollment in 2023, continuing to invest in CMC and then data readouts and ultimately, a registration. It also opens up capital allocation to drug discovery, as Gilmore also mentioned, and additional in vivo therapeutics. So given the unfriendly state of the equity markets, we feel really comfortable that this cash extension will really put us in a good position for once we get to the value inflection points [indiscernible].

Brian Cheng

Maybe one last question, maybe for Gilmore or Michelle. What are the key data catalysts that — or milestone that you see for your company in the next 12 months that investors should focus on?

Gilmore O’Neill

Well, I think that — and Michelle, I’ll hand to you in a second if you want to add to that, I think the key milestones will be as I outlined. I think there are some strategic deliverables. I think we need to bring in that CSO, and I think people will have to get to know the CSO. But sort of the key ones I think I would focus on right now would be in our 301 data readouts, if you’re looking for those immediate catalysts in the short term, and obviously, our execution and then our delivery of data in the middle and the end of this year. And then finally, obviously, as we reboot discovery, our CSO joins, we look forward to being able to share more about our targets and — beyond HSCs. But I think, as I said, the key catalyst really in the near term this year will be around our 301 data readouts, and we’re really excited about this program.

Brian Cheng

Great. I think that’s — we’re at the top of our 20 minutes together. Thank you so much for joining us. That concludes the presentation and Q&A today. Thank you.

Gilmore O’Neill

Thank you.